Study of Safety and Dosing Effect on SMN Levels of Valproic Acid (VPA) in Patients With Spinal Muscular Atrophy
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ClinicalTrials.gov Identifier: NCT00374075 |
Recruitment Status
:
Completed
First Posted
: September 8, 2006
Last Update Posted
: August 23, 2016
|
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Spinal Muscular Atrophy | Drug: Valproic Acid | Phase 1 |
Study Type : | Interventional (Clinical Trial) |
Enrollment : | 42 participants |
Allocation: | Non-Randomized |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | In Vivo Study of Safety, Tolerability and Dosing Effect on SMN mRNA and Protein Levels of Valproic Acid in Patients With Spinal Muscular Atrophy |
Study Start Date : | September 2003 |
Study Completion Date : | February 2006 |

- To assess safety and tolerability of VPA in SMA patients greater than 2 years of age
- To look for a potential in vivo effect of VPA on SMN mRNA in patient blood cells at routinely used clinical doses
- Measures of gross motor function
- Electrophysiologic measures of denervation
- DEXA estimates of body composition, bone mineral density and content
- Measures of pulmonary function

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Ages Eligible for Study: | 2 Years and older (Child, Adult, Senior) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Patients must have a diagnosis of SMA, confirmed by genetic testing
- Only patients 2 years of age and older at enrollment will be eligible
Exclusion Criteria:
- Patients taking any medications with known hepatotoxicity, congenital metabolic disorders or on multiple anticonvulsant medications
- Patients taking medications which may interact with VPA
- Patients on ventilatory support for more than 16 hours per day
- Patients currently enrolled in other treatment trials

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00374075
United States, Utah | |
University of Utah/Primary Children's Medical Center | |
Salt Lake City, Utah, United States, 84132 |
Principal Investigator: | Kathryn J Swoboda, M.D | University of Utah/Primary Children's Medical Center |
Additional Information:
Publications:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: | Kathryn Swoboda, M.D., University of Utah |
ClinicalTrials.gov Identifier: | NCT00374075 History of Changes |
Other Study ID Numbers: |
11893 |
First Posted: | September 8, 2006 Key Record Dates |
Last Update Posted: | August 23, 2016 |
Last Verified: | August 2016 |
Keywords provided by Kathryn Swoboda, University of Utah:
Spinal Muscular Atrophy (SMA) Valproic Acid |
Additional relevant MeSH terms:
Atrophy Muscular Atrophy Muscular Atrophy, Spinal Spinal Cord Diseases Pathological Conditions, Anatomical Neuromuscular Manifestations Neurologic Manifestations Nervous System Diseases Signs and Symptoms Central Nervous System Diseases Motor Neuron Disease Neurodegenerative Diseases |
Neuromuscular Diseases Valproic Acid Anticonvulsants Enzyme Inhibitors Molecular Mechanisms of Pharmacological Action GABA Agents Neurotransmitter Agents Physiological Effects of Drugs Antimanic Agents Tranquilizing Agents Central Nervous System Depressants Psychotropic Drugs |